SAFETY

WAINUA was generally well tolerated1

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Results from the NEURO-TTRansform trial

Vitamin A warning: Treatment with WAINUA leads to a decrease in serum vitamin A levels. Patients should supplement with the recommended daily allowance of vitamin A while taking WAINUA. Refer patients to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.*1

  1. *Higher doses than the recommended daily allowance of vitamin A should not be given to try to achieve normal serum vitamin A levels during treatment with WAINUA, as serum vitamin A levels do not reflect the total vitamin A in the body.1

ADVERSE REACTIONS IN ≥5% OF PATIENTS TREATED WITH WAINUA1

Adverse reaction WAINUA (N=144)
Vitamin A decreased† 15%
Vomiting 9%
Proteinuria 8%
Injection-site reactions‡ 7%
Blurred vision 6%
Cataract 6%

  1. †Vitamin A decreased includes vitamin A deficiency and vitamin A decrease.1

  2. ‡Injection-site reactions include erythema, pain, and pruritus.1

  • Three serious adverse reactions of AV heart block (2%) occurred in WAINUA-treated patients, including 1 case of complete AV block1
  • Safety and tolerability profile of WAINUA was consistent at Weeks 66 and 852

No new safety signals were identified through 3 years of treatment initiation3

  INDEX STUDY: WAINUA
(N=144)		 EXTENSION STUDY: WAINUA
(N=127)
Exposure, mean (SD), days 540.8 (82.18) 626.2 (209.4)
Serious TEAE* 27 (19) 34 (27)
Serious TEAE considered possibly related to study drug by investigator 0 1 (1)
TEAE leading to death 3 (2) 6 (5)
AESI: Thrombocytopenia 3 (2) 5 (4)
AESI: Glomerulonephritis 0 0

*TEAE is defined as the AE that first occurred or worsened after the first dose of WAINUA in the extension or index study.3

All data are n (%) unless otherwise stated.

The fatal TEAEs in the extension study were not considered to be related to WAINUA by the investigator or sponsor.3

Among the five patients with AESIs of thrombocytopenia, one experienced an SAE that was not considered to be related to WAINUA by the investigator or sponsor. The remaining four patients had non-serious AEs that were considered possibly related or related to WAINUA.3

Discover the efficacy of WAINUA

WAINUA efficacy

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Learn more about once-monthly self-administration

Dosing & administration

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FOOTNOTES AND ABBREVIATIONS

AE, adverse event; AV, atrioventricular; AESI, adverse events of special interest; SAE, serious adverse event; SD, standard deviation; TEAE, treatment emergent adverse event.

REFERENCES

  1. 1. WAINUA® (eplontersen) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2025.
  2. 2. Coelho T, Marques W Jr, Dasgupta NR, et al. Eplontersen for hereditary transthyretin amyloidosis with polyneuropathy [article and supplementary online content]. JAMA. 2023;330(15):1448-1458.
  3. 3. Berk J, Coelho T, Conceição I, et al. Long-term efficacy and safety of eplontersen in patients with hereditary transthyretin amyloidosis with polyneuropathy: initial report from the open-label extension of the NEURO-TTRansform study. Presented at: 5th International ATTR Amyloidosis Meeting 2025; September 25-26, 2025; Baveno, Italy.